A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

Recruiting now

Conditions studied: Beta-Thalassemia

In brief

The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.

Key facts

Study ID: NCT06271512
Run by: Genetix Biotherapeutics Inc.
People needed: 150
Starts: 2024-01-23
Expected to finish: 2043-12-01
Last updated by the study team: 2025-02-11

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.
Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations.
Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).
Participant must be followed by a hematologist based in the US.

You may not qualify if…

There are no exclusion criteria for Registry participation.

Where it is running

UCSF Benioff Children's Hospitals — Oakland, California, United States (enrolling)
Stanford University — Palo Alto, California, United States (enrolling)
University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
Cohen Children's Medical Center — New Hyde Park, New York, United States (enrolling)
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
Texas Children's Hospital — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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